Jump to content
  • Listening to the patient saved many lives

    Article information
    • USA
    • Blogs
    • Pre-existing
    • Original author
    • No
    • Vincent Rajkumar
    • 10/02/21
    • Everyone


    Dr S. Vincent Rajkumar is a Professor of Medicine at the Mayo Clinic in Rochester, Minnesota. In this account, originally published via a Twitter thread, Dr Rajkumar remembers how the insight of Mike Katz, a patient with myeloma, left an incredible legacy for patient safety.


    I would like to share the story of how a patient with cancer came up with the idea for a randomised trial, and how listening to him saved a lot of lives.

    In 2002, I had just completed a randomised trial with the notorious drug thalidomide for the cancer, multiple myeloma. Thalidomide would later be FDA approved on the basis of this trial. As a young investigator I was thrilled with the success and eager for the next exciting trial testing fancy new regimens.

    But a patient with myeloma, Mike Katz, had other ideas. 

    Mike was on national patient advocacy committees. He had battled myeloma for years and knew all of the recent advances. More importantly he attended numerous patient support group meetings and had his finger on the pulse of what myeloma patients were going through.

    Mike was also on the ECOG-ACRIN Cancer Research Group and the National Cancer Institute myeloma committee and listened as we debated ideas for the next myeloma trial.

    While doctors talked about creating “exciting” combinations, Mike said, “Listen, what patients really want is freedom from the side effects of Dexamethasone.”

    He said, “All these new drugs don’t help if patients cannot take them. You guys are giving too much Dexamethasone. And people are suffering.”

    Dexamethasone was used in myeloma at high doses to kill the cancer cells. It was an important component of therapy.

    Mike disagreed.

    “You are giving Dexamethasone at a high dose on the basis that this is how it has always been done. Please run a trial and see if in the era of new drugs you still need such high doses of Dexamethasone.”

    We were all sceptical. But Mike was not going to give up. He insisted we do a randomised trial of high dose Dexamethasone versus low dose dexamethasone.

    To us the idea seemed destined to fail. It seemed so boring. We had waited 40 years for new drugs and Mike wants us to test Dexamethasone  dosing!

    However, we respected Mike. We knew he was aware of what patients were going through. We saw 100-200 myeloma patients a year. He interacted with thousands. He was also leading meetings of support group leaders who were leading meetings with lots of other myeloma patients.

    So we proceeded to convince the National Cancer Institute and ECOG-ACRIN Cancer Research Group leadership that testing the optimal dose of Dexamethasone was the most important publicly funded randomised trial. Rafael Fonseca took the lead.

    It wasn’t easy. But we got it approved.

    Long story short, the trial accrued faster than any other myeloma trial we had done in national cooperative groups ever!

    Deaths with high dose Dexamethasone (control, standard of dare arm) were significantly higher than with low dose dexamethasone!

    We had hypothesised that by using low dose Dexamethasone we will have less toxicity and similar efficacy. Little did we know that just a change in Dexamethasone dose would save lots of lives: At one year 96% were alive with low dose Dexamethasone  versus 87% with high dose standard of care Dexamethasone.

    There were other benefits as expected. All serious side effects including blood clots were lower with low dose Dexamethasone.

    The Lenalidomide plus low dose Dexamethasone (Rd) regimen was born. The little “d” signifies low dose Dexamethasone.

    Rd is now the backbone of most myeloma regimens. The lower dose of Dexamethasone has allowed us to build many 3-4 drug combinations.

    We are indebted to Mike. We grieve his loss. His legacy and work with ECOG-ACRIN Cancer Research Group, the American Society of Clinical Oncology (ASCO), the National Cancer Institute, the National Institutes of Health and the International Myeloma Foundation endures.

    The ASCO honored Mike in 2014 with the Partners in Progress Award. He narrated this story when he accepted the Award at the ASCO Annual Meeting.   

    Our randomised trial of high dose versus low dose Dexamethasone was published in The Lancet Oncology and is one of the most cited myeloma papers ever with over 1000 citations.

    Here is his son Jason sharing his father‘s story.

    0 reactions so far


    Recommended Comments

    There are no comments to display.

    Create an account or sign in to comment

    You need to be a member in order to leave a comment

    Create an account

    Sign up for a new account in our community. It's easy!

    Register a new account

    Sign in

    Already have an account? Sign in here.

    Sign In Now
  • Create New...